Monday, March 24, 2014

I have a hard time leaving the fate of my kid up to researchers. I know and appreciate that a ton of great research is being done to find new and novel treatment options to help patients with Cystic Fibrosis, but it's not happening fast enough and that's not okay with me. Two years ago, when I attended my very first NACFC, I found somewhere deep within the program book of abstracts ad article on Gallium and the potential for its use in treating pseudomonas infections. Six months later, at an unrelated meeting in DC, I heard the president of the CFF mention Gallium as something that would probably be a pretty good thing for CF patients...in the future. Fast forward to 2013 when Drew acquired Achromobacter, the miserable multi-drug resistant bacteria that has been causing him problems for over a year now. One of the first thoughts that I had was "gallium". Then, this past fall when I attended the 2013 NACFC, Gallium was presented during one of the Plenary Sessions. I must try gallium.I'm no scientist. I'm sort of like a doctor with far less textbook training but way more real life experience. (I kid! (sort of)) My understanding of the way gallium works in CF is this: When a bacteria like pseudomonas starts to grow in your airways, it binds together creating something called a biofilm, which makes it incredibly hard for antibiotics to penetrate. Achromobacter, like pseudomonas, also creates a biofilm. It is even more resistant to antibiotics than pseudomonas. In fact, the kind Drew has is completely resistant to all antibiotics. Studies have show a 16% decline in lung function ever 48mo when a patient is colonized with achromobacter. On one hand that sucks and is terrifying. On the other hand, this is just a research paper. Either way, this is not okay with me. I started to push the envelope a little, emailing Drew's doctor more, asking everyone I know who has more medical knowledge than me about gallium and it's uses and its potential, inside and outside of CF. I've asked about dosing and forms (inhaled vs injected) and how I can get my hands on it to try it. The information that I found on the current clinical trials for gallium are all looking at the bacteria pseudomonas. They're also suggesting that the estimated study completion date is September 2018. That is more than 4 years from now! That's 4% for us. And that also is the study on pseudomonas. I completely understand and appreciate that steps are taken to understand the safety and efficacy of different drugs before they become available to patients, but there has got to be a better way. This drug is already available to patients for a number of different things. We need to find ways to work together and use the testing that they've already done that brought this drug through FDA approval to lessen the time that it would take for others to benefit from it's use. I don't know if gallium (aka Ganite) will help Drew, but I'm going to find out. If you're a doctor or scientist whose reading this, tell me what you know about Ganite. Help me to piece these things together to discover who in this community could benefit from this drug. Are there different ways to administer it that could limit any harmful effects? I am playing scientist now and I need people to help me collect everything available on this drug so that I can make sense out of it. It's easy for a doctor or researcher to say to me "just be patient" when it's not their kids life on the line. I know that there are tremendous new therapies available and soon to be available to CF patients and I pray that Drew will find benefit from them. However, I need him to be well when he starts them. My job as his parent is to keep him alive and well, and I will do that. I cannot sit back and wait for someone else to determine the fate of my child. I will talk about this and talk about this and talk some more, collecting resources, making connections, finding what I need to either prove or eliminate this drug as a reasonable treatment option for my kid, not because I want to stir the pot or because I don't have anything better to do, but because his life depends on it. His life depends on me and I'm going to find a way to save him. I will leave you with this, stolen shamelessly from another fantastic CF blog. "My heart reminded me the race in the battle against cf is NOT a sprint based upon numbers. It’s a MARATHON of decisions based upon individual knowledge, circumstances and needs." That's the truth. It's exhausting, fighting this way, but when you have no choice, hope keeps you going.

Last week I traveled to Washington, DC for two meetings. One was on the important role that patients & caregivers plan in understanding medication adherence, and the other was on patients and caregivers being involved as disruptive innovators in healthcare; both topics very close to my heart. While I was reimbursed for my travel expenses (less alcohol), I was not compensated for my time. I struggle with this. Part of me gets angry because my time is valuable, to a lot of people, and I want these folks to see and appreciate that. The other part of me wants to be heard, no matter the compensation.
During one of the meetings, I participated as a panelist and shared on the role that I play on our healthcare team. Also on our panel was the amazing Emily (@emilykg1), an adult CF patient and passionate advocate. I view myself as a partner, and I work with different tools that our doctor has recommended (medications, medical devices) and with tools that I've found or created to manage Drew's health as best I can. I think that our message was well received, that by partnering in care, meeting patients where they are rather than where you want them to be, has meaning. And patients who are meaningfully engaged in their own care have a better chance at a good outcomes.
One of the final questions that the audience asked us was what ideas we have for getting more people like us involved at every level, and I countered with a question. I asked this audience, made up of doctors and other clinicians, pharmacists, admirals and even representatives from the Surgeon Generals office, how many of them had to use vacation time to attend the meeting today. No one raised their hand....except for Emily. Patients and caregivers are asked to give up time in their already full and complicated schedules to help out these healthcare professionals. Meaningful engagement would be asking patients to be a part of the team, recognizing the value that they bring to the table, and showing them that you appreciate them through respect and compensation.
Last week, after scanning receipts and filling out forms for reimbursement, I got the most amazing email:

"I wanted to reach out to you to let you know that your comments regarding your participation at our meeting being not part of your ‘job’ did not fall on deaf ears. In addition to your time at the meeting, you also spent a lot of time planning prior to the meeting and working with us after the meeting to work through your expense reimbursement. Our team would like to compensate you for your time.

In addition, one of our Co-Investigators put forth a very thoughtful suggestion to propose to the Steering Committee, a policy to compensate patients/patient advocates/caregivers (not paid by an organization) when they participate in one of our events. If the Steering Committee agrees to this policy change, our program as a whole will not overlook compensating important contributions of patients and caregivers at future meetings.

To be compensated for my time is great. For my message to be heard, for others to recognize and appreciate the value of patients and caregivers sitting at the table for these critical discussions that will be fundamental to the changing culture of healthcare, that is an amazing feeling. I have the utmost respect for this organization, and hope that their actions will be seen and replicated by others.

Tuesday, March 4, 2014

I had a really incredible week last week, first in DC and then in NYC. I was in DC for the second meeting of the Patient Engagement Committee at the Cystic Fibrosis Foundation. The CFF really did an outstanding job gathering this group of patients, parents, doctors & other clinicians, pharmacy reps, insurance companies and drug companies to work together on solutions for enhancing the relationship that each has with the patient to improve outcomes and save lives. I enjoy all things related to these meetings. The second trip I made last week was to NYC for the kick off meeting of their Clinical Data Research Network. They have brought together the brightest and the best in NYC from 5 of the major medical institutions to share ideas and data to create a network of robust clinical and patient reported data that will help to improve both health and care across the city. Ultimately, this network should serve as a model for the rest of the country and the value of collaboration and data transparency will become evident to all. When the meeting kicked off, the principal investigator put a picture of her 18mo old twins on the screen. She said that every morning she asks herself if all of the work that she is doing is more important that spending time with her babies. And while it is definitely a balance, she believes that the work she is doing will be so instrumental in changing healthcare for the better that she makes the sacrifice. I thought about the question for a little bit. It's not easy for our family when I am working at Children's or taking phone calls at all hours of the day. When I do travel for a night or two, it sends our family into a little tailspin as they try to figure out where the lady who runs the show has gone. My amazing husband does a fantastic job of keeping everything in line when I am away, but everyone is out of sorts when one of us isn't there. We are a team, the 6 of us. And I thought about whether or not all that I do is worth it, worth leaving my family for, for an hour or a day or even just a dinner. The answer for me is yes, it's worth it because this is what is going to save his life. Some other random thoughts about my days....I have good days and I have bad days (more good than bad), but I always keep going, trying to figure out what can be done so that I don't lose him. In January when we were getting bad test results, I was running out the door when I slipped on some ice and bit it in my driveway. I wanted to scream and curse the world but I had to keep going. After school, everyone is tired and crying, wanting drinks and snacks and attention. I must get a breathing treatment started. I must call the pharmacy and the hospital to update our insurance information. In perpetual motion, I step on a plug. OUCH!!!! Again, I want to cry and curse this cruel world but I have to keep on going. Start dinner, do homework, bathe children. Everyday at 3pm I put on my shoes and some old jeans, a free fleece that I "won" fundraising. I'd love to look nicer like the other moms at pick-up. I wish I had time to do my hair and makeup, money to buy fashionable clothes, cute boots. But I spend most of my money on a babysitter while I'm out shouting from the rooftops "We have to do better. We have to move faster. What can I do so that I don't lose him?!" I dig hopelessly through a bag of candy hearts while running out that door at 3pm, because when I give my 5yr old a candy heart that says "My Girl" on it in the lobby of her school, the smile that overtakes her tired little face makes my day better. We have to leave for Girl Scouts. Only one has shoes on. Only one is missing pants. Luckily no one has to get out of the car for the quick drop before we return home to pull together a dinner and then head back out to pick her up because it's only 19 degrees out and those chubby pantless legs have got to be cold. Sometimes I think we look homeless - mismatched clothes, hair uncombed. Our house usually looks like squatters have inhabited it. Do I wish it was cleaner? Hell yeah I do! But this girl is out of time and energy, and at the end of the day there are just more important things in life. As we sit down to eat, the phone rings. Vietnam Vets, wondering if we have anything to donate. "Yes!! Bring your truck, come in and take whatever you want, spring cleaning simplified", I think. I agree to a donation, but then think about when I will find the time to dig through the ruins to pull out a bag of toys that we don't use anymore, or a pile of clothes that we've outgrown. I'll find something for them. When I have to say no to a social event or a fundraiser or any other kind of fun activity in my life it's definitely not because I don't want to do it. I want to go out with friends. I want to have some drinks, stay out too late, laugh. But being less than 100% when I wake up the next morning sets me back more than just that one day.So much of what I do really is stuff that I want to do. I love doing what I'm doing, and I wish I could do so much more. I hate having to prioritize treatments and appointments and phone calls over the fun stuff, but we do what we have to do for our family right now. I know that was very random, but just wanted to get some thoughts out. Oh yeah, and my heart is still beating for anyone who cares. More cardiology follow up in the next week(s) but until then we will just keep on keepin' on.

"65 Roses"is what some children with cystic fibrosis call their disease because the words are much easier for them to pronounce.

This blog, 66 Roses, is dedicated to finding the cure.

Cystic fibrosis is a life shortening genetic disease that affects the lungs and digestive system of about 30,000 children and adults in the United States (70,000 worldwide). Thick mucus clogs the lungs and leads to life threatening infection. The pancreas is also obstructed by this thick mucus production, imparing digestion and leading to malnutrition. - www.cff.org